Generic Drug May Ease Autism in Children

Dec. 11, 2012 -- An inexpensive generic drug may ease autism in children, a small new study shows.

The drug, bumetanide, is a diuretic, or a drug that rids the body of extra water through urine. It’s been FDA-approved since 1983 to reduce fluid buildup in patients with heart failure, kidney disease, or liver disease.

In autism, bumetanide may help to correct a chemical imbalance in the brain, says Jing-Qiong Kang, MD, PhD, an assistant professor of neurology at Vanderbilt University in Nashville, Tenn. Kang studies the biology of autism, but she was not involved in the current research.

Unlike other medications, which are used to quell behavioral problems that are sometimes associated with autism, like hyperactivity, insomnia, and aggression, bumetanide seems to ease some of the core features of the condition, including problems connecting and interacting with other people.

“They were making more eye contact, more spontaneous speech, and more two-way conversation,” says Daniel Coury, MD, a pediatric psychiatrist in Columbus, Ohio, who is medical director for the Autism Speaks Autism Treatment Network.

“At the end of the day parents said, ‘I like this. My child is doing better,’” says Coury, who was not involved in the research.

The Research

For the study, French researchers had 60 children with autism take either bumetanide or a look-alike placebo pill for 90 days.

Children in the study ranged from 3 to 11 years old. Their symptoms were scored by clinicians who did not know whether they were taking the drug or the placebo. Parents and teachers were also asked about changes in the children’s behavior over time.

By the end of the study, children in the treatment group shifted from average autism scores in the severe range to the mild or moderate range.

“A third of the placebo group improved, but three-fourths of the treatment group improved. So that suggests that it seems to be a real finding there,” Coury says.

The study is published in the journal Translational Psychiatry.

More Research Needed

Despite the hopeful news, Coury and other experts urged caution.

“Is it going to have a sustained improvement, a continued improvement? There’s a lot that we don’t know,” he says.